Focus on... DIAN Tests Treatments for Early-Onset Alzheimer's

In early 2013, the Dominantly Inherited Alzheimer's Network Trials Unit (DIAN TU) at Washington University School of Medicine in St. Louis will begin testing three investigational drugs in 160 volunteers with inherited mutations for early-onset Alzheimer’s. Each of the test drugs appears to either reduce production of beta amyloid or clear it from the brain. Based on their family history, the volunteers are within 10 to 15 years of the age when symptoms of cognitive decline and dementia are expected to appear. They will be randomly assigned to receive one of three investigational drugs (75 percent chance) or a placebo (25 percent chance). The trial also will monitor the health of 80 participants without the mutation, who will receive a placebo.

Earlier DIAN studies have shown that people who have Alzheimer's mutations are likely to have biological indicators that show the disease is beginning in the brain, including evidence of brain plaques by PET amyloid imaging and changes in specific protein levels in cerebrospinal fluid, years before the appearance of clinical symptoms. Scientists will monitor these biomarkers of early Alzheimer's to test whether the treatments slow or stop the disease process.

The first part of the trial is planned to last 2 years but may be extended and expanded if one or more of the drugs prove to be effective in slowing or stopping the disease. The trials unit is supported by the NIA-funded DIAN; the Alzheimer's Association; and the DIAN Pharma Consortium, composed of 10 pharmaceutical companies. The makers of the drugs are supplying them free of charge and are providing financial support for the trial. To learn more about the trial, go to www.dianexpandedregistry.org/AboutUs.htm.